OLIGONUCLEOTIDES TARGETING GAL3ST1

The present invention relates to antisense oligonucleotides (oligomers) complementary to GAL3ST1 pre-mRNA sequences, which are capable of inhibiting the expression of GAL3ST1. Inhibition of GAL3ST1 expression is expected to be beneficial for the treatment of metachromatic leukodystrophy. La présente...

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Hauptverfasser:	FRYLAND, Tue, HAGEDORN, Peter, RASMUSSEN, Søren Vestergaard, LERBECH JENSEN, Marianne
Format:	Patent
Sprache:	eng ; fre
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	The present invention relates to antisense oligonucleotides (oligomers) complementary to GAL3ST1 pre-mRNA sequences, which are capable of inhibiting the expression of GAL3ST1. Inhibition of GAL3ST1 expression is expected to be beneficial for the treatment of metachromatic leukodystrophy. La présente invention concerne des oligonucléotides (oligomères) antisens complémentaires de séquences de pré-ARNm GAL3ST1, qui sont aptes à inhiber l'expression de GAL3ST1. L'inhibition de l'expression de GAL3ST1 devrait être bénéfique pour le traitement de la leucodystrophie métachromatique.